Ultragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $203 Million
Key Highlights
- ➤Ultragenyx agrees to sell rare pediatric disease PRV for $203 million
- ➤FAYUVI became the first FDA-approved treatment for MPS IIIA
- ➤PRV transaction closing awaits customary conditions, including Hart-Scott-Rodino waiting-period expiration
- ➤Proceeds strengthen non-dilutive funding and support Ultragenyx’s path to profitability
Expert Statements
Howard Horn, Chief financial officer and executive vice president, corporate strategy at Ultragenyx
“The sale of this PRV delivers meaningful non-dilutive funding that strengthens our ability to provide innovative treatments for rare and ultra-rare diseases while supporting our objective of achieving profitability”
Howard Horn, Chief financial officer and executive vice president, corporate strategy at Ultragenyx
“FAYUVI itself benefited from capital generated from a previous PRV sale, demonstrating the important role the PRV program plays in helping companies develop transformative therapies for rare disease patients.”
NOVATO, Calif., Oct. 14, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $203 million. Ultragenyx received the PRV upon U.S. Food and Drug Administration (FDA) approval of FAYUVI™ (rebisufligene etisparvovec-hopf), also known as UX111, the first-ever FDA-approved treatment for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A).
“The sale of this PRV delivers meaningful non-dilutive funding that strengthens our ability to provide innovative treatments for rare and ultra-rare diseases while supporting our objective of achieving profitability,” said Howard Horn, chief financial officer and executive vice president, corporate strategy. “FAYUVI itself benefited from capital generated from a previous PRV sale, demonstrating the important role the PRV program plays in helping companies develop transformative therapies for rare disease patients.”
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